A specialist pharmacist is calling on the government to consider modern compounding as part of the solution to the cost of orphan drugs
Gold Coast pharmacist Matthew Bellgrove has highlighted that the cost of these drugs in the treatment of rare diseases is escalating.
He says that if eyes turned to the compounding sector, there could be significant savings for families as well as the government’s Life Saving Drugs subsidy program.
“There are some orphan drugs that can, and should, only be sourced through the manufacturer who spent many years developing them,” Mr Bellgrove says.
“However there are evidence-based treatments that can be safely and legally compounded here in Australia in government-approved compounding laboratories for a fraction of the cost.
“Governments could then encourage drug companies to develop medicines for other rare conditions that don’t yet have a truly effective treatment.”
Mr Bellgrove pointed to an article published in the Journal of Clinical Pharmacy and Therapeutics, which called into question the cost-effectiveness of drug companies being encouraged to develop orphan drugs when much cheaper alternatives could be sourced.
In the article titled, “Do we need authorized orphan drugs when compounded medications are available?”, Dr Steven Simoens and a team of researchers from the Research Centre for Pharmaceutical Care and Pharmaco‐economics in Belgium argued that orphan drugs should not be authorised for funding by drug administration bodies when alternatives such as compounded drugs and off-label medication were available on the market at a reasonable cost.
“A better balance should be struck between the development of orphan drugs along the recently established regulatory pathways and the pragmatic use of pharmacy‐compounded products and evidence‐based off‐label use of already available commercial products,” Dr Simoens and his team wrote.
“Societal needs would be best met by focusing orphan drug development on rare diseases for which there is a high unmet medical need.”
Mr Bellgrove also pointed to a paper published in 2015 in the Journal of Clinical Pathology also questions the cost-effectiveness of the current orphan drug authorisation regime.
Researchers from the University of Oxford, Centre for Evidence-Based Medicine and University of Washington in Seattle found that Pedea, used to treat a vascular condition in preterm infants costs 82 000 times as much as generic ibuprofen, “yet, research evidence has shown that Pedea is not superior to oral ibuprofen in clinical effectiveness…”
The authors of the paper go on to say, “The available evidence suggests that there is inconsistency in the quality of evidence of approved orphan drugs, and there is no clear mechanism for determining their prices. In some cases, far cheaper generic agents appear to be available. A more robust, transparent and standard mechanism for determining annual costs is imperative.”